VIII因子半衰期产品：真实世界经验。

FVIII half-life products: A real-world experience.

作者信息

Zanon Ezio, Porreca Annamaria, Napolitano Angela, Simion Chiara, Simioni Paolo

机构信息

Haemophilia Center, First Chair of Internal Medicine, Via Giustiniani 2, University Hospital of Padua, 35128 Padua, Italy.

Department of Human Sciences and Promotion of the Quality of Life, Unit of Clinical and Molecular Epidemiology IRCCS San Raffaele Roma Open University, 00166 Rome, Italy; Unit of Clinical and Molecular Epidemiology IRCCS San Raffaele Roma, Rome, Italy.

出版信息

Thromb Res. 2025 May;249:109306. doi: 10.1016/j.thromres.2025.109306. Epub 2025 Mar 14.

DOI:10.1016/j.thromres.2025.109306

PMID:40147314

Abstract

BACKGROUND

Haemophilia A is a genetic coagulation disorder requiring prophylactic Factor VIII (FVIII) treatment to prevent joint damage and enhance quality of life. The introduction of extended half-life (EHL) FVIII products represents a major advancement, addressing patient needs for reduced infusion frequency, improved efficacy, and enhanced safety.

METHODS

This single-centre observational study examined the real-world use of FVIII products among 124 male patients treated at the Haemophilia Centre of Padua from January 2018 to December 2023. Data on patient characteristics, treatment regimens, and pharmacokinetic profiles were analyzed.

RESULTS

Among the FVIII products used, Advate®, Elocta®, and Jivi® showed median half-lives of 11.25 [10.75-12.25], 16.50 [13.75-17.75], and 15.38 [13.38-18.63] hours, respectively. Esperoct® exhibited the longest half-life at 19.75 [16.00-24.50] hours, enabling reduced infusion frequency. EHL products showed significantly lower weekly infusion rates compared to standard half-life (SHL) products (1.4 [1.0-2.0] vs. 2.0 [2.0-3.0], p < 0.001), with comparable bleeding control among the different FVIII-EHL.

CONCLUSIONS

EHL FVIII products offer significant clinical benefits, reducing the burden of frequent infusions and improving adherence while maintaining effective bleeding control. Despite these advancements, comprehensive evaluations of cost, safety, and long-term outcomes are essential to optimize their integration into haemophilia care.

摘要

背景

甲型血友病是一种遗传性凝血障碍疾病，需要预防性使用凝血因子 VIII（FVIII）进行治疗，以预防关节损伤并提高生活质量。延长半衰期（EHL）FVIII 产品的推出是一项重大进展，满足了患者减少输注频率、提高疗效和增强安全性的需求。

方法

这项单中心观察性研究调查了 2018 年 1 月至 2023 年 12 月在帕多瓦血友病中心接受治疗的 124 名男性患者对 FVIII 产品的实际使用情况。分析了患者特征、治疗方案和药代动力学概况的数据。

结果

在所使用的 FVIII 产品中，Advate®、Elocta® 和 Jivi® 的中位半衰期分别为 11.25 [10.75 - 12.25]、16.50 [13.75 - 17.75] 和 15.38 [13.38 - 18.63] 小时。Esperoct® 的半衰期最长，为 19.75 [16.00 - 24.50] 小时，能够降低输注频率。与标准半衰期（SHL）产品相比，EHL 产品的每周输注率显著更低（1.4 [1.0 - 2.0] 对 2.0 [2.0 - 3.0]，p < 0.001），不同 FVIII - EHL 产品在出血控制方面相当。